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# Priority Review for Genentech sNDA for Zelboraf
- URL: https://www.fdaweb.com/priority-review-for-genentech-snda-for-zelboraf/
- Published: 2017-08-09T12:00:00.000Z
- Updated: 2026-09-14T22:40:01.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139348

FDA has accepted for priority review a Genentech supplemental NDA for Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD is a rare, serious blood disease characterized by the abnormal multiplication of certain white blood cells called histiocytes. The therapy was also granted breakthrough therapy designation for the indication. A user fee review target date has been set for 12/7.

The submission includes data from the open-label, Phase 2 VE-BASKET study, which the company says included an innovative clinical trial design that helped collect data faster and may accelerate the development of medicines for diseases with high unmet need. “Instead of enrolling people based primarily on their disease or its location, basket studies match a disease’s underlying genetic profile to the mechanism of action of the medicine,” Genentech says. This allows researchers to evaluate multiple diseases simultaneously.

  
The BASKET study investigated the use of Zelboraf for people with BRAF V600 mutation-positive cancers and other diseases, including ECD. Final results for the 22 people with ECD showed a best overall response rate of 54.5%, the company says. “The median duration of response, progression-free survival and overall survival were not reached at a median follow-up time of 26.6 months,” it adds.